News

Astria Therapeutics is the new name of the company that’s developing STAR-0215, an investigational kallikrein-inhibitor previously known as QLS-215, for the treatment of hereditary angioedema (HAE). Formerly called Catabasis Pharmaceuticals, the company said it changed its name to reflect its pledge of having patients serve as its guiding…

Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…

Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older. That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo —…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…

The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people with hereditary angioedema (HAE). KalVista said it is working closely with study investigators and clinical sites so…

The United Arab Emirates (UAE) has approved BioCryst Pharmaceuticals’ Orladeyo (berotralstat) for preventing hereditary angioedema (HAE) attacks in patients, 12 and older. “As the first targeted oral, once-daily treatment, Orladeyo provides an important new treatment option for patients and physicians,” Henrik Balle Boysen, executive vice president and chief operating…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

The prices of three approved preventive treatments — Cinryze, Haegarda, and Takhzyro (lanadelumab) — for hereditary angioedema (HAE) must be reduced by more than half for these medications to be cost-effective. Those are the conclusions of a new report published by the Institute for Clinical and Economic Review,…

Regulatory health agencies in Canada and Switzerland are reviewing BioCryst Pharmaceuticals’ applications for the approval of Orladeyo (berotralstat) in their countries for preventing hereditary angioedema (HAE) attacks in patients ages 12 and older. The applications — to Heath Canada and Swissmedic — followed Orladeyo’s recent approvals in the…